FDA approves treatments for heart failure caused by rare disease

Health Problems

(HealthDay)—Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) capsules have been approved to treat adults with cardiomyopathy caused by transthyretin mediated amyloidosis (ATTR-CM), the U.S. Food and Drug Administration announced today. Recommended dosage is four 20-mg capsules of Vyndaqel once daily or a single 61-mg capsule of Vyndamax once daily, according to the manufacturer. The two drugs, which are the first approved […]

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FDA approves drugs for treatment of two rare blood diseases

Health Problems

(HealthDay)—Two drugs have been approved by the U.S. Food and Drug Administration for the treatment of rare blood diseases, the agency announced Friday. Elzonris (tagraxofusp-erzs) infusion was granted the first approval for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and children aged 2 years and older. Researchers investigated the efficacy of Elzonris in two patient cohorts […]

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US approves new drug for prevention of migraines

Health Problems

Migraine sufferers on Thursday got some relief from US regulators who approved the first of a new class of drugs for migraine prevention. The United States Food and Drug Administration said it endorsed Aimovig for prevention of migraine in adults, through monthly self-injections. Aimovig is the first in a new class of drugs that work by blocking the activity of […]

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FDA approves CAR T therapy for large B-cell lymphoma

Health Problems

The U.S. Food and Drug Administration (FDA) has expanded approval for a personalized cellular therapy developed at the University of Pennsylvania’s Abramson Cancer Center, this time for the treatment of adult patients with relapsed or refractory large B-Cell lymphoma after two or more lines of systemic therapy. Today’s approval includes treatment of diffuse large B-cell lymphoma (DLBCL) – the most […]

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